Digna Biotech SL

The general aim of the study is to determinate safety, tolerability and early pharmacokinetics of cardiotrophin.1 in healthy volunteers.

This is a Phase I trial aimed to determine the safety of the investigational gene therapy product (rAAV2/5-PBGD) for the treatment of Acute Intermittent Porphyria (AIP).
Up to eight patients fulfilling the eligibility criteria will participate in this multicentre, open label, single dose, dose-ranging Phase I clinical trial.
The enrolled patients will be followed up to assess the safety profile of the investigational gene therapy product and to establish the maximum therapeutic safe dose to be administered in future confirmatory/pivotal clinical trial(s). In addition, the biological and clinical response to the treatment with rAAV2/5-PBGD in AIP patients will be assessed.
A complete evaluation of the clinical (symptoms and quality of life assessment) and laboratory (blood and urine) data will be performed.